A Study of the Safety of REN001 in Patients With Primary Mitochondrial Myopathy

Overview

The purpose of this study is to assess REN001 safety in subjects with primary mitochondrial myopathy

Full Title of Study: “An Open-label Study to Evaluate the Safety and Tolerability of 12 Weeks Treatment With Oral REN001 in Patients With Primary Mitochondrial Myopathy (PMM), With an Optional Extension of Treatment”

Study Type

  • Study Type: Interventional
  • Study Design
    • Allocation: N/A
    • Intervention Model: Single Group Assignment
    • Primary Purpose: Treatment
    • Masking: None (Open Label)
  • Study Primary Completion Date: April 23, 2020

Interventions

  • Drug: REN001
    • Once daily

Arms, Groups and Cohorts

  • Experimental: Group
    • REN001 Low Dose

Clinical Trial Outcome Measures

Primary Measures

  • Adverse Events
    • Time Frame: Comparing Baseline to Week 12
    • Number of participants with Adverse Events as a measure of safety and tolerability

Secondary Measures

  • Adverse Events
    • Time Frame: Continous to Week 48
    • Number of participants with Adverse Events as a measure of safety and tolerability

Participating in This Clinical Trial

Inclusion Criteria

  • Subjects must give written, signed and dated informed consent
  • Confirmed diagnosis of PMM according to the 2016 Rome Consensus recommendations
  • Confirmed mitochondrial mutation with evidence of myopathy
  • Able to remain on stable medication throughout the study

Exclusion Criteria

  • Documented evidence of ongoing rhabdomyolysis
  • Subjects with motor abnormalities other than related to mitochondrial disease
  • Treatment with an investigational drug within 3 months prior to Day 1
  • Hospitalised within 3 months prior to screening for any major medical condition
  • Pregnant or nursing females

Gender Eligibility: All

Minimum Age: 16 Years

Maximum Age: N/A

Are Healthy Volunteers Accepted: No

Investigator Details

  • Lead Sponsor
    • Reneo Pharma Ltd
  • Provider of Information About this Clinical Study
    • Sponsor
  • Overall Official(s)
    • Grainne Gorman, MD, Principal Investigator, Wellcome Centre for Mitochondrial Research

Clinical trials entries are delivered from the US National Institutes of Health and are not reviewed separately by this site. Please see the identifier information above for retrieving further details from the government database.

At TrialBulletin.com, we keep tabs on over 200,000 clinical trials in the US and abroad, using medical data supplied directly by the US National Institutes of Health. Please see the About and Contact page for details.